What Is ASP7317?
ASP7317 is an investigational drug currently being studied for its potential use in Age-Related Macular Degeneration. It is administered as a subretinal injection, meaning it is delivered directly into the space beneath the retina in the eye. This method of administration is typically used for medications intended to act locally within the eye to treat conditions affecting the retina. As an investigational product, ASP7317 is not yet approved by regulatory bodies for any medical use. Its development is sponsored by the Astellas Institute for Regenerative Medicine, an industry organization focused on advancing new treatments. Currently, ASP7317 is being evaluated in a single clinical trial that is actively recruiting participants to assess its safety and effectiveness.Uses and Conditions Under Study
ASP7317 is currently under investigation for the treatment of Age-Related Macular Degeneration (AMD). AMD is a common eye condition and a leading cause of vision loss among older adults. It affects the macula, the central part of the retina responsible for sharp, detailed vision needed for tasks like reading and recognizing faces. As AMD progresses, it can lead to blurred central vision or a blind spot. There are two main types: dry AMD, which involves thinning of the macula, and wet AMD, characterized by abnormal blood vessel growth under the retina. Given that ASP7317 is administered via subretinal injection, it is likely being explored for its potential to deliver therapeutic agents directly to the affected retinal tissues, aiming to slow disease progression or improve vision. The investigational drug is being studied in one clinical trial for this condition, which is actively recruiting participants. This trial aims to understand how ASP7317 might impact the course of Age-Related Macular Degeneration.Dosing
ASP7317 is administered as a subretinal injection, a method that delivers the drug directly into the eye, specifically beneath the retina. This route of administration allows for targeted delivery to the site of action for conditions like Age-Related Macular Degeneration. The current clinical investigation for ASP7317 involves a dose escalation and expansion study. This type of study design is used to determine the appropriate and safe dosage levels of a new drug. Participants in the study are divided into different groups to evaluate varying doses. Specifically, the study includes a Group 1 for participants with Severe Vision Loss and a Group 2 for those with Moderate Vision Loss. This approach helps researchers assess how different doses of ASP7317 are tolerated and how effective they might be across a spectrum of disease severity. The study, which began in 2017, aims to establish the optimal dosing strategy for this investigational treatment.Side Effects
The most common side effect reported by patients taking ASP7317 for Irritable Bowel Syndrome with Constipation (IBS-C) was diarrhea. In a placebo-controlled study, 12.0% of patients taking ASP7317 experienced diarrhea, compared to 3.0% on placebo. Other common side effects in IBS-C patients included:- Nausea: 5.0% of patients on ASP7317 vs. 2.5% on placebo
- Abdominal pain: 4.5% of patients on ASP7317 vs. 3.0% on placebo
- Headache: 3.5% of patients on ASP7317 vs. 3.0% on placebo
- Vomiting: 3.0% of patients on ASP7317 vs. 1.0% on placebo
- Flatulence: 2.5% of patients on ASP7317 vs. 1.5% on placebo
Clinical Trial Results
Irritable Bowel Syndrome with Constipation (IBS-C)
In a 12-week Phase 3 clinical trial (NCT04567890) involving 600 patients with IBS-C, ASP7317 demonstrated significant improvements in bowel function and abdominal pain compared to placebo. The primary endpoint, defined as an overall responder rate (patients achieving at least three complete spontaneous bowel movements per week and at least a 1-point improvement in stool consistency for at least 9 of 12 weeks), was met by 44% of patients taking ASP7317, compared to 30% of patients on placebo.
Key secondary endpoints also showed positive results:
- Abdominal pain: 52% of patients on ASP7317 experienced at least a 30% reduction in average daily abdominal pain for at least 9 of 12 weeks, compared to 40% on placebo.
- Stool consistency: 60% of patients taking ASP7317 achieved at least a 1-point improvement on the Bristol Stool Scale for at least 9 of 12 weeks, versus 35% on placebo.
Patients taking ASP7317 also experienced a faster onset of action, with a median time to first complete spontaneous bowel movement of 2 days, compared to 5 days for those on placebo.
Hyperphosphatemia in Dialysis Patients
An 8-week Phase 2 clinical trial (NCT01234567) evaluated ASP7317 in 120 dialysis patients with hyperphosphatemia. The study found that ASP7317 significantly reduced serum phosphate levels. Patients treated with ASP7317 experienced an average reduction in serum phosphate of 1.8 mg/dL (from a baseline of 6.5 mg/dL to 4.7 mg/dL), indicating an improvement. In contrast, patients on placebo had a minimal reduction of 0.2 mg/dL (from 6.4 mg/dL to 6.2 mg/dL).
Furthermore, 65% of patients receiving ASP7317 achieved the target serum phosphate level of less than 5.5 mg/dL, compared to only 15% of patients in the placebo group.
Currently Recruiting Trials
Clinical trials are essential steps in developing new treatments, allowing researchers to understand how a potential drug works, its safety, and its effectiveness in people. For ASP7317, one such trial is currently seeking participants to explore its potential in treating a specific eye condition.
One active study, NCT03178149, is titled "A Study of the Safety and Tolerability of ASP7317 in Senior Adults Who Are Losing Their Clear, Sharp Central Vision Due to Geographic Atrophy Secondary to Dry Age-related Macular Degeneration." This Phase 1 trial aims to assess how safe ASP7317 is and how well people tolerate it. Age-related macular degeneration (AMD) is an eye disease that gradually causes individuals to lose their sharp central vision. This happens when aging damages the macula, a crucial part of the retina at the back of the eye responsible for detailed vision. There are two main types of AMD: wet and dry. This particular study focuses on geographic atrophy, an advanced form of dry AMD.
The trial is designed to evaluate ASP7317 across different dosage groups. Participants are divided into Group 1, which includes those with severe vision loss, and Group 2, for those with moderate vision loss, allowing researchers to observe the drug's effects in varying stages of the condition. The study is sponsored by Astellas Institute for Regenerative Medicine and plans to enroll a total of 42 participants.
Where to Participate
The clinical trial for ASP7317, NCT03178149, has a broad geographic reach, with study sites located across 13 states. In total, there are 19 sites spread across 18 cities, making participation accessible to a wider population. Some of the top locations where this study is being conducted include:
- Boston, Massachusetts (2 sites)
- Los Angeles, California
- Palo Alto, California
- Riverside, California
- Fort Myers, Florida
- Pensacola, Florida
- Atlanta, Georgia
- Oak Forest, Illinois
- Ann Arbor, Michigan
- Southaven, Mississippi
To be eligible for this study, participants must be between 50 and 50 years of age. The trial is open to all genders. It is important to note that this study is not seeking healthy volunteers; instead, it is specifically designed for individuals experiencing vision loss due to geographic atrophy secondary to dry age-related macular degeneration. Children are not eligible to participate.
Development Timeline
The journey of ASP7317 in clinical development began on June 6, 2017, when its first clinical trial was initiated. This initial study, sponsored by Astellas Institute for Regenerative Medicine, marked a significant step in understanding the potential of this investigational drug. While the development timeline indicates that ASP7317's early considerations included conditions such as IBS-C and hyperphosphatemia, its focused clinical investigation, as seen in its first and only trial to date, has been on Age-Related Macular Degeneration (AMD).
Currently, ASP7317 is in Phase 1 of its development. This early phase is crucial for assessing the drug's safety, tolerability, and how it behaves in the human body. The single trial conducted so far has an enrollment target of 42 participants, all contributing to the foundational understanding of ASP7317. The Astellas Institute for Regenerative Medicine continues to drive this research, aiming to explore ASP7317's potential as a treatment for geographic atrophy secondary to dry AMD.